Roche & Isis Join Forces on Antisense Drug for Huntington’s Disease

Xconomy San Diego — 

San Diego’s Isis Pharmaceuticals (NASDAQ: ISIS) and Roche, the Swiss Pharmaceuticals giant) have formed an alliance to develop new treatments for Huntington’s disease, according to a statement released today. Roche agreed to pay Isis $30 million upfront, with potential revenue from licensing and milestone payments totaling $362 million. In addition, Isis would get tiered royalties on potential drug sales.

Isis, which is actually based north of San Diego in Carlsbad, CA, specializes in antisense therapies, developing drugs that target harmful proteins produced by mutated genes that are responsible in certain types of disease. By synthesizing a strand of nucleic acid that binds to the mutated gene’s messenger RNA (mRNA), antisense drugs block protein production.

There is currently no effective treatment or cure for Huntington’s disease, an inherited genetic brain disorder that results in the progressive loss of both mental faculties and physical control. The goal of current therapies is to slow symptoms, which include behavioral changes, abnormal movements, and dementia, and to help patients function for as long and as comfortably as possible.

Huntington’s occurs in about five to 10 out of every 100,000 people from Western Europe. It is estimated that about 30,000 people in the United States are affected. Symptoms usually appear between the ages of 30 to 50, and progressively worsen over a 10- to 25-year period.

Initially, research will focus on Isis’ lead drug candidate that blocks production of all forms of the huntingtin (HTT) protein, the protein responsible for Huntington’s disease.

In a statement, Isis COO B. Lynne Parshall says, “We believe that Roche’s expertise in developing CNS drugs, along with their clinical development experience, will greatly enhance our development efforts for this program and allow us to move forward more rapidly. In addition, by utilizing Roche’s brain shuttle technology with our antisense drug discovery capabilities, we have the potential to significantly improve the therapeutic potential for this program.”